HEMGENIX offers elevated and sustained factor IX
levels for years after a single infusion1
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Review product-specific resources for the first gene therapy for hemophilia B.
Four-year data now available
Take a closer look at HEMGENIX efficacy, safety, and durability at 4 years.1
Learn moreAbout hemophilia B
Hemophilia B is a rare, X-linked hereditary disorder characterized by bleeding, pain, and long-term complications.2-4 Hemophilia B is caused by a defect in a gene encoding coagulation factor IX, which is expressed primarily in hepatocytes. This mutation occurs in 5 per 100,000 male births.4-6 The severity of hemophilia B typically correlates to native coagulation factor activity. Severe hemophilia B is characterized by spontaneous and/or traumatic bleeding into joints, muscles, and internal organs and can result in reduced life expectancy.3,4,7
What is HEMGENIX?
HEMGENIX is an adeno-associated virus vector-based gene therapy indicated for the treatment of adults with hemophilia B (congenital factor IX deficiency) who:
• Currently use Factor IX prophylaxis therapy, or
• Have current or historical life-threatening hemorrhage, or
• Have repeated, serious spontaneous bleeding episodes.
HEMGENIX is for single use intravenous infusion only.
Please see full prescribing information for HEMGENIX.
What are the key benefits?
At 4 years, a single dose of HEMGENIX was associated with:
REDUCTION IN FACTOR
IX CONSUMPTION1
MEAN FACTOR
IX ACTIVITY1
REDUCTION IN
ANNUALIZED BLEED
RATE (ABR)1
VS ROUTINE FACTOR
IX PROPHYLAXIS*
At 4 years, HEMGENIX continues to:
Have NO reported serious treatment-related adverse reactions1
NOT be associated with the development of inhibitors to factor IX8
*Adjusted ABR for all bleeds decreased from an average of 4.16 for prophylaxis during the lead-in period to 1.63 in months 7 to 48 after treatment, a reduction of 61%. During months 7 to 18, ABR for all bleeds decreased by 64%.
Explore value perspectives
Get payer and manufacturer perspectives on how to evaluate breakthrough treatments like HEMGENIX in the report The Value of Innovative Medicine.
Read nowThe value of HEMGENIX
For a 1-million-member plan, it is estimated that 2 patients may qualify for HEMGENIX.† According to ICER’s analysis, HEMGENIX was projected to be a dominant treatment compared with factor IX prophylaxis with lower costs and higher quality-adjusted life years. ICER’s base-case analysis projected HEMGENIX use would result in >$6 million in savings over the lifetime of a hemophilia B patient compared with the standard of care.9
Abbreviation: ICER, Institute for Clinical and Economic Review.
†Approved indication may also include people who are not currently on prophylaxis. See approved prescribing information for appropriate patient selection.
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